Drug proven to slow progression of rare, fatal lung disease may also reduce the risk of death

Newly published study supports demand for publicly-funded access to only approved treatment for idiopathic pulmonary fibrosis

MARKHAM, ON, May 23, 2014 /CNW/ - Newly published data is giving Canadian patients suffering from mild to moderate forms of idiopathic pulmonary fibrosis (IPF) hope that publicly-funded access to the only approved treatment option may soon be a reality. The Phase 3 ASCEND Study: A Randomized, Double-Blind, Placebo-Controlled Trial of Pirfenidone in Patients with Idiopathic Pulmonary Fibrosis (IPF), was presented at the 2014 International Conference of the American Thoracic Society (ATS) in San Diego, California, and concurrently published in The New England Journal of Medicine. The much anticipated study results confirm the proven efficacy and safety of Esbriet® (pirfenidone), the first and only treatment approved in Canada for IPF – a rare, progressive and ultimately fatal lung disease.

According to the published data, the Phase 3 ASCEND study's primary endpoint demonstrated that Esbriet significantly reduced the decline in lung function by 47.9 per cent, as measured by a decline in forced vital capacity of 10 per cent or more. Additionally, the study showed that 27.5 per cent of patients on Esbriet experienced a reduced decline of the 6-minute walk distance (6MWD), and almost half (43 per cent) saw an improvement in progression-free survival (PFS) with IPF. Importantly, a pooled analysis of data from ASCEND and two previous phase 3 trials (CAPACITY) with Esbriet showed that the risk of all-cause mortality in IPF patients was reduced by 48 per cent compared with placebo.

"As we've seen in the recent articles published in the New England Journal of Medicine and presented at ATS, the ASCEND data further reinforces the extensive body of clinical evidence and international expert consensus supporting the use of Esbriet for adults with a diagnosis of mild to moderate IPF," said Dr. Shane Shapera, respirologist at Toronto General Hospital, University Health Network. "Also, the results of the INPULSIS study provide evidence that another agent, nintedanib, may add a second drug to our arsenal against this terrible disease. These results are major breakthroughs and I urge the government to review the evidence and re-evaluate their decision on public funding of Esbriet for patients with IPF."

New data demands immediate access for Canadian patients

The initial elation of Canadian patients at hearing that the first-ever treatment for IPF had been approved by Health Canada turned to dismay when they learned that access to this potentially life-extending medication was being denied by public funders. Despite disease severity, the absence of other treatment options, and a priority review by Health Canada, the Common Drug Review (CDR) recommended that public drug programs not list Esbriet due to a 'lack of evidence,' despite governments of more than 13 European countries providing public funding based on the same data. The resulting lack of government funding for Esbriet has been especially devastating for seniors who are most affected by this disease, as they rely primarily on public drug programs.

"The ASCEND trial data has given the IPF community renewed hope – it's like a breath of fresh air for those who have been desperately awaiting the results of this study," said Robert Davidson, president and founder of the Canadian Pulmonary Fibrosis Foundation (CPFF). "The fact that Esbriet has now been confirmed to not only slow disease progression, but also to reduce the risk of death, means that the provinces and territories must heed the evidence from ASCEND and make Esbriet immediately accessible to patients whose lives depend on it."

Some IPF patients in Canada have lost their battle with the disease while fighting for access to the only treatment that could have extended and improved their lives. Even with all-party support from elected officials in many provinces including Ontario, government decision-makers have refused to grant IPF patients access to a drug prescribed as medically necessary by doctors who are experts in this rare disease.

"It has been extremely disheartening to see our government leaders turn their backs on us at our greatest time of need, as we watch members of our community decline and succumb to this horrible disease," said Barbara Barr, an IPF patient from King City, Ontario. "Our resolve has been greatly bolstered by the publication of this new data in one of the world's leading medical journals – weighty evidence that no government decision-maker can or should ignore."

About IPF in CanadaIdiopathic Pulmonary Fibrosis (IPF) is a rare, progressive and fatal lung disease that has no known cause. It is characterized by scarring of the lungs, which hinders a patient's ability to breathe. Up to 30,000 Canadians are believed to be affected by all forms of PF, with an estimated 3,000 to 5,000 suffering from mild to moderate IPF. Typically diagnosed later in life, IPF has a life expectancy of just two to five years, which is similar to many other malignant diseases, including lung, breast and colon cancer. However, some rapidly progressing cases can be lethal within months of diagnosis.

About the Canadian Pulmonary Fibrosis Foundation The Canadian Pulmonary Fibrosis Foundation (CPFF) is a registered not-for-profit charitable organization established to provide support, hope and resources for those affected by pulmonary fibrosis. Robert Davidson, president of the CPFF, who had IPF and received a double lung transplant in January 2010, founded the organization in 2009 to help educate others, and to answer those non-medical questions frequently asked by those suffering with the disease. For more information, please visit www.cpff.ca.